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Pannobhaso
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A researcher into gene silencing as a technology for improving the lives of those suffering from incurable diseases. Benitec Biopharma is my primary investment in this technology as ddRNAi offers one-time treatments for a broad range of these diseases.
  • Another DdRNAi Program To Be In The Clinic In 2015 19 comments
    Jul 11, 2014 1:21 PM | about stocks: BNIKF, BTEBY

    Researchers from the University of Sheffield in the UK are now predicting that a ddRNAi treatment for Motor Neurons Disease (MND), also known as Amyotrophic Lateral Sclerosis (ALS), will be ready for regulatory approval by August 2015. They say, "The experience that we have accumulated with our gene therapy and Clinical Trials programmes at SITraN is crucial to develop this new therapy for patients with ALS/MND. We plan submission for regulatory approval by August 2015, for permission to take this therapy to patients in the clinic. "

    While this article does not say this work is ddRNAi, a little research reveals that Professor Mimoun Azzouz, who is mentioned in the article, published a paper back in 2006 on the subject. Furthermore, the ALS Advocate refers to Professor Pamela Shaw's work at University of Sheffield's Institute for Translational Neuroscience (SITraN) and her work is in silencing the SOD1 gene using AAV9 delivery of SOD1-shrna. These references confirm that this research is ddRNAi.

    This team better get its skates on though because teams from the Research Institute at Nationwide Children's Hospital and the Ludwig Institute at the University of California, San Diego, are doing exactly the same work in their labs and they have already conducted studies in NHP.

    It would seem that in a little over 12 months we could have another ddRNAi program or two moving into the clinical stage of development.

    Both teams operate under the safe harbor provision of international patents and so no payments are due to Benitec (BNIKF, BTEBY) before commercialisation but the fact that the technology is progressing to clinical trial outside of Benitec's own pipeline is a big plus for the technology.

    Disclosure: The author is long BNIKF.

    Additional disclosure: This article is not intended as investment advice. Readers should due their own research.

    Themes: ALS, MND, shrna, Benitec, SOD1 Stocks: BNIKF, BTEBY
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Comments (19)
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  • omcdac
    , contributor
    Comments (124) | Send Message
     
    Great info. Another DdRNAi Program is coming soon.
    Soon benitec will take over ALNY.

     

    Thanks
    Pannobhaso
    11 Jul, 03:28 PM Reply Like
  • brama66
    , contributor
    Comments (98) | Send Message
     
    My sentiments about ALNY exactly, omc! Go benitec!
    11 Jul, 07:45 PM Reply Like
  • TiersMonde
    , contributor
    Comments (5) | Send Message
     
    Great find. However, I wonder if Benitec's patents cover all indications. Is there anyone with insight into this?
    13 Jul, 01:21 PM Reply Like
  • Pannobhaso
    , contributor
    Comments (162) | Send Message
     
    Author’s reply » These Instablogs may be of interest.

     

    http://seekingalpha.co...

     

    http://seekingalpha.co...
    14 Jul, 02:15 AM Reply Like
  • timfrisbie
    , contributor
    Comments (13) | Send Message
     
    Enjoy reading your Benitec articles. I'm starting to wonder though why RA Capital just sold a million or so shares and the one board member retired. Is there something that we, the investors, are missing that others are seeing? To me, this has the possibilities of being a 15 billion dollar company in a few short years, we'll worth the risk IMO. Obviously we all know the worst case scenario's, but Pannabhosa, what are your 1,3, and 5 year predictions/guesses?
    Thank you
    14 Jul, 02:39 PM Reply Like
  • timfrisbie
    , contributor
    Comments (13) | Send Message
     
    Pannabhosa, I appreciate your articles on Benitec. The market cap remains so low compared to competitors and with RA Capital just dumping approx 1 million shares, is there something we're missing? In my opinion, if things pan out this could be a 15 billion dollar company in a few short years. What are your thoughts, predictions on share price over next 2-5 years? Obviously we are all aware of the worst case scenerio's, but what do you attribute the dismal sp/ market cap and lack of interest to?
    14 Jul, 02:40 PM Reply Like
  • Pannobhaso
    , contributor
    Comments (162) | Send Message
     
    Author’s reply » Hi Timfrisbie, the director who resigned was always going to retire. He resigned from other directorships last year. There really is no story here.

     

    RA Capital did take some profit but their motives are not clear and so I am unable to comment. However, as they still retain the majority of their holdings, I can only assume that they remain confident in their position.

     

    In other posts, I have addressed the matter of Benitec's market capitalization compared to its competitors and I won't go over that material again but there certainly is a difference. When Benitec can secure a deal with a major pharmaceutical company, in the way that companies like Alnylam and ISIS have done, then the pps will more accurately reflect Benitec's true position in the RNAi/gene therapy world.

     

    The next 2-5 years will be the most exciting time in the company's history, however, they could also be the most volatile. Success in biotech is never assured and so it is important that a company like Benitec has more than one iron in the fire. Benitec's diverse pipeline and partnering programs will, IMO, pay off over the next 5 years, regardless of what happens in the next 12 months. That said, I will leave the pps guesswork to those with a better crystal ball than I.

     

    I would like to think that the TT-034, HCV program will be successful as that would be the fastest way to have the pps take off but I am realistic enough to understand that outcomes for HCV drugs are notoriously difficult to predict and achieve. I think the NSCLC program has great promise and I look forward to that going into the clinic in 2015. The AMD program represents the next generation of ddRNAi technology and so it has a pivotal role to play in the company. It may also be the dark horse in the pipeline by attracting an investment from big pharma before its more advanced pipeline siblings.

     

    I think there is a genuine need for the permanent silencing of genes in the treatment of many indications. As such, ddRNAi will have a rosy future if the company can demonstrate success through one of these programs that I have referred to.
    14 Jul, 09:49 PM Reply Like
  • GreenGrowthGeek
    , contributor
    Comments (736) | Send Message
     
    More solid information Panno. Thank you. I guess the market is over reacting again to the resignation/retirement of a director and the RA Capital sale. I am down about 40% on my holdings so I hope we get some positive movement soon. I love the company but it has killed my portfolio in the past 5 months.
    15 Jul, 12:37 PM Reply Like
  • petethepanzer
    , contributor
    Comments (1042) | Send Message
     
    theres no mention of ddrnai or benitec in the article

     

    therefore it is probably not ddrnai
    17 Jul, 06:42 AM Reply Like
  • Pannobhaso
    , contributor
    Comments (162) | Send Message
     
    Author’s reply » The previous references to professors Shaw and Azzouz work both demonstrate that they are using shrna, so I think we can be sure that the work is ddRNAi.
    17 Jul, 10:33 AM Reply Like
  • petethepanzer
    , contributor
    Comments (1042) | Send Message
     
    if this was true why would benitec not gloat about it in their company presentation

     

    sounds like it kills the rest of their pipeline and they would surely own a piece of it
    19 Jul, 10:34 AM Reply Like
  • Pannobhaso
    , contributor
    Comments (162) | Send Message
     
    Author’s reply » Hi Peterthepanzer, there are literally hundreds of of labs around the world using ddRNAi/shrna under the safe harbor provisions of national and international patents. It is impossible for the company to track them all and, even if it could, there would be little point because no license is required until the research is turned into a commercial product. This means that a research team or another company could take a ddRNAi drug all the way through Pl/Pll/Plll trials and still not have to pay Benitec one cent for the use of the technology.

     

    I am sure that Benitec will be very interested in this work as a piece of research and further validation of their technology but one must also keep in mind that the SOD1 gene is only associated if around 25% of familial ALS sufferers, which, in turn, only make up 10% of all ALS cases. This means that the commercial potential of this research is quite limited and so it is not quite the "pipeline killer" that you may think it is.
    19 Jul, 12:06 PM Reply Like
  • petethepanzer
    , contributor
    Comments (1042) | Send Message
     
    10% is still huge as a market consider PTC Therapeutics their drug for als only treats the 15% with nonsense mutations

     

    sarepta their current als drug only treats 13% of patients the rest of their exon targets would cover more but their mostly very early all preclinical

     

    how do these labs get access to ddrnai before the company knows about it is there no recipe isnt there some complexity to creating the rnai cassette that the company themself would have to instruct you on
    19 Jul, 01:03 PM Reply Like
  • Pannobhaso
    , contributor
    Comments (162) | Send Message
     
    Author’s reply » "isnt there some complexity to creating the rnai cassette that the company themself would have to instruct you on"

     

    The technology is widely described in scientific journals so no instruction is required from Benitec. This is no different to siRNA. There are lots of companies and universities using siRNA (Dicerna, Arrowhead, etc.) but they don't need Alnylam or U Mass to tell them how to do it.

     

    With regard to ALS, the SOD1 gene target DOES NOT represent 10% of ALL sufferers. 10% of ALS sufferers are familial (90% are sporadic). Of the 10% familial sufferers ONLY 25% of them are associated with the SOD1 gene.
    19 Jul, 10:41 PM Reply Like
  • petethepanzer
    , contributor
    Comments (1042) | Send Message
     
    ok so its only 2.5% well then your correct

     

    im wondering does ddrnai cause cells to age faster it does not seem natural for the cells to produce thousands of extra rnai products for their lifetime
    20 Jul, 09:24 AM Reply Like
  • Pannobhaso
    , contributor
    Comments (162) | Send Message
     
    Author’s reply » "im wondering does ddrnai cause cells to age faster it does not seem natural for the cells to produce thousands of extra rnai products for their lifetime"

     

    As far as I am aware, there is no evidence to indicate any change in the aging process of a transduced cell.
    20 Jul, 09:36 PM Reply Like
  • petethepanzer
    , contributor
    Comments (1042) | Send Message
     
    ok how can a cell be instructed to produce this much extra rna product (thousands more) for the life of the cell and not be using up alot of its energy doing so...this is my concern here
    21 Jul, 08:50 AM Reply Like
  • Pannobhaso
    , contributor
    Comments (162) | Send Message
     
    Author’s reply » I will not speculate on this. As I said, I am not aware of any research which points to the cell aging process being accelerated because of ddRNAi. If you find some research which supports this theory, then I would be very happy to read it.

     

    One thing that you may also want to consider is that, while "extra" RNA may be being produced in the targets cells, because shrna turns off the production of proteins, these same cell are now producing less proteins. Which is the more energy intensive mechanism?
    21 Jul, 09:30 AM Reply Like
  • petethepanzer
    , contributor
    Comments (1042) | Send Message
     
    unless it was hep c which was not supposed to be there in the first place...i suppose hep c already answers the question since livers dont neccessarily age faster if the viral load is high
    21 Jul, 10:42 AM Reply Like
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